Exploring Long-Chain Fatty Acid Oxidation Disorders: Recognizing the Intricacies in Disease Management, the Available Agents in Treatment, and Considerations for Pharmacists Across Practice Settings

This activity was originally broadcast live at the 2022 Asembia Specialty Pharmacy Summit.

This activity features a Caregiver Perspective.

Program Description

Long-chain fatty acid oxidation disorders (LC-FAODs) are classified as rare, life-threatening, autosomal recessive genetic disorders characterized by acute crises of energy production and chronic energy deficiency. The recent approval of an oral liquid as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed LC-FAODs offers the first and only dietary intervention for these patients. As integral members of the health care team, it is imperative that specialty and managed care pharmacists are knowledgeable about the pathophysiology, clinical presentation, diagnostic criteria, and incidence of LC-FAODs as well as the role of this emerging therapy in addressing clinical manifestations and impact that LC-FAODs have on patient health-related quality of life. Recognizing this disorder will enable pharmacists across practice settings to implement early intervention strategies to efficaciously manage, reduce, or prevent occurrence of serious metabolic crises and complications associated with LC-FAODs. This activity will also include a patient interview to provide a better understanding of the management of these rare disorders and their impact on the lives of patients and caregivers.

Target audience: Specialty pharmacists and managed care pharmacists

Type of activity: Application
Release date: May 10, 2022
Expiration date: May 10, 2023

Time to complete activity: 1.5 hours
Fee: Free

Educational Objectives

At the completion of this activity, participants will be able to:

• Analyze the pathophysiology and clinical presentation associated with the most common long-chain fatty acid oxidation disorders (LC-FAODs) as well as the significant disease-related complications and impact on patient quality of life
• Explain the limitations of current standards of care for managing LC-FAODs and the safety and efficacy data for available therapies, including dosing, titration, and administration of the available agent
• Explore the roles of specialty pharmacists and managed care pharmacists as clinicians and patient educators in improving access to novel therapies for management of LC-FAODs